Ocugen - Model OCU410 - Modifier Gene Therapy Vaccine
OCU410 (AAV-RORA) is a modifier gene therapy product candidate being developed for the treatment of dry age-related macular degeneration (Dry AMD). OCU410 utilizes an AAV delivery platform for the retinal delivery of the RORA (RAR Related Orphan Receptor A) gene. Various genes associated with AMD are regulated by RORA. The RORA protein plays an important role in lipid metabolism and demonstrates an anti-inflammatory role, which we believe could be a potential therapeutic candidate for dry AMD.
Dry AMD affects approximately nine to ten million patients in the United States and there is currently no approved treatment for the disease. AMD is a degeneration of the macula of the retina that leads to impairment and loss of central vision. AMD is characterized by thickening and loss of normal architecture within Bruch’s membrane, lipofuscin accumulation in the retinal pigment epithelium (“RPE”), and drusen formation beneath the RPE in Bruch’s membrane. These deposits consist of complement components, other inflammatory molecules, lipids, lipoproteins B and E, and glycoproteins. Dry AMD involves the slow deterioration of the retina with submacular drusen, atrophy, loss of macular function and central vision impairment. Currently, there is no approved treatment for dry AMD, which accounts for 85-90% of the total AMD population.